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An Efficacy, Safety, Tolerability, Pharmacokinetics and Pharmacodynamics Study of BIIB067 (Tofersen) in Adults With Inherited Amyotrophic Lateral Sclerosis (ALS)

NCT02623699 · Status: COMPLETED · Phase: PHASE3 · Type: INTERVENTIONAL · Enrollment: 176

Last updated 2023-07-28

Study results available
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Summary

The primary objectives of Parts A and B of this study are to evaluate the safety, tolerability, and pharmacokinetics (PK) of ascending doses of tofersen in adults with ALS and a documented superoxide dismutase 1 (SOD1) mutation. The primary objective of Part C of this study is to evaluate the clinical efficacy of tofersen administered to adults with ALS and a confirmed SOD1 mutation.

The secondary objective of Parts A and B of this study is to evaluate the effects of tofersen on levels of total SOD1 protein in the cerebrospinal fluid (CSF). The secondary objectives of Part C are to evaluate the safety, tolerability, pharmacodynamic (PD), and biomarker effects of tofersen.

Conditions

Interventions

DRUG

Tofersen

Administered as specified in the treatment arm.

DRUG

Placebo

Administered as specified in the treatment arm.

Sponsors & Collaborators

  • Ionis Pharmaceuticals, Inc.

    collaborator INDUSTRY

  • Biogen

    lead INDUSTRY

Principal Investigators

  • Medical Director · Biogen

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Model
PARALLEL

Eligibility

Min Age
18 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2016-01-20
Primary Completion
2021-07-16
Completion
2021-07-16
FDA Drug
Yes

Countries

  • United States
  • Australia
  • Belgium
  • Canada
  • Denmark
  • France
  • Germany
  • Italy
  • Japan
  • Poland
  • South Korea
  • United Kingdom

Study Locations

More Related Trials

Entities

Drugs
Diseases
Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT02623699 on ClinicalTrials.gov